First bid to use CRISPR to edit human genes up for federal review

A federal panel will get its first look next week at a proposal to use the revolutionary gene-editing technology CRISPR in humans, in this case to try to treat cancer.

The experiment, proposed by the University of Pennsylvania, would remove certain cells of the immune system, called T cells, from cancer patients. Scientists would then use CRISPR to genetically modify the T cells so that when they’re returned to the patient, they target and destroy myeloma, melanoma, and sarcoma tumor cells.

Carrie Wolinetz, the associate director for science policy at the National Institutes of Health, disclosed the review in a blog post, which was first reported by MIT Technology Review. The federal panel must review all human experiments that alter DNA.

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A biotech company, Editas Medicine in Cambridge, Mass., had been expected to be the first to use CRISPR in people, in an effort to treat a rare eye disease. But it looks like Penn might beat Editas to the punch.

Penn scientist Dr. Carl June pioneered the use of genetically modified T cells, called CAR-T’s, as a cancer treatment. So far, researchers have used traditional genetic engineering to make the cells home in on the molecules that stick out of tumor cells. But that’s less efficient and often less precise than CRISPR.

Penn is reportedly seeking NIH approval to use CRISPR to edit out two genes in T cells. One, called PD-1, suppresses the ability of T cells to attack tumors. The other is a T cell receptor that can turn the body’s natural defenses against itself.

The medical significance of the proposal is not clear, since gene editing has already been used in humans — just not gene editing via the CRISPR-Cas9 system.

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A different genome-editing technique, called TALEN, was used to modify T cells to treat a British toddler with leukemia last year. That was the first time TALEN had been used to treat a patient. A third genome-editing technique, called zinc fingers, is in an advanced clinical trial sponsored by California-based biotech Sangamo BioSciences to treat AIDS, with promising results in more than 80 patients.

Big money has lined up behind each of the rival genome-editing techniques. Seattle’s Juno Therapeutics promised Editas as much as $737 million to use its CRISPR technology in CAR-T research, while Novartis entered a $280 million alliance with Intellia Therapeutics.